ClinicalTrials.gov
ClinicalTrials.gov Menu

Autologous Stem Cell Treatment for Chronic Lung Disease Study

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03044431
Recruitment Status : Completed
First Posted : February 7, 2017
Results First Posted : October 25, 2017
Last Update Posted : October 15, 2018
Sponsor:
Information provided by (Responsible Party):
Melissa Rubio, PhD, APRN, Lung Institute

Study Type Observational
Study Design Observational Model: Cohort;   Time Perspective: Prospective
Conditions COPD
Interstitial Lung Disease
Intervention Procedure: Cell therapy
Enrollment 207
Recruitment Details All patients who underwent elective, investigational cell therapy were enrolled in the outcomes study.
Pre-assignment Details No patients in the study received placebo. All patients received autologous, adult stem cell therapy.
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Period Title: Overall Study
Started 207
Completed 148
Not Completed 59
Reason Not Completed
Lost to Follow-up             59
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Overall Number of Baseline Participants 207
Hide Baseline Analysis Population Description
All patients who underwent cell therapy were included in the data analysis.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 207 participants
<=18 years
0
   0.0%
Between 18 and 65 years
42
  20.3%
>=65 years
165
  79.7%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 207 participants
Female
95
  45.9%
Male
112
  54.1%
Race and Ethnicity Not Collected   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants
[1]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Number of Participants with Chronic Obstructive Pulmonary Disease (COPD)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 207 participants
167
  80.7%
Number of Participants with Interstitial Lung Disease (ILD)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 207 participants
40
  19.3%
Treatment Type  
Measure Type: Count of Participants
Unit of measure:  Participants
Venous Protocol Number Analyzed 207 participants
159
  76.8%
Double Venous Protocol Number Analyzed 207 participants
26
  12.6%
Bone Marrow Protocol Number Analyzed 207 participants
22
  10.6%
Baseline FEV1 if participant has COPD   [1] 
Mean (Full Range)
Unit of measure:  FEV1 Percentage (%)
Number Analyzed 167 participants
36.9
(9 to 85)
[1]
Measure Analysis Population Description: 167 patients in the overall sample were diagnosed with chronic obstructive pulmonary disease (COPD).
Baseline Clinical COPD Questionnaire Score All Participants   [1] 
Mean (Full Range)
Unit of measure:  Units on a scale
Number Analyzed 207 participants
3.6
(0.8 to 6.0)
[1]
Measure Description: The Clinical COPD Questionnaire (CCQ) is a 10-item scale where the participant chooses responses on a 0-6 scale for each item. 0 represents no symptoms and 6 represents severe symptoms. The mean of the responses is the final score and ranges from 0-6, 0 is least severe symptoms and 6 is most severe symptoms.
COPD Participant Baseline QOL (quality of life) score based on the Clinical COPD Questionnaire (CCQ)   [1] [2] 
Mean (Full Range)
Unit of measure:  Units on a scale
Number Analyzed 167 participants
3.6
(1.0 to 6.0)
[1]
Measure Description: The Clinical COPD Questionnaire (CCQ) is a 10-item scale where the participant chooses responses on a 0-6 scale for each item. 0 represents no symptoms and 6 represents severe symptoms. The mean of the responses is the final score and ranges from 0-6, 0 is least severe symptoms and 6 is most severe symptoms.
[2]
Measure Analysis Population Description: 167 patients in the overall sample had a diagnosis of COPD.
ILD Participant Baseline QOL score based on Clinical COPD Questionnaire (CCQ)   [1] [2] 
Mean (Inter-Quartile Range)
Unit of measure:  Units on a scale
Number Analyzed 40 participants
3.3
(0.8 to 5.6)
[1]
Measure Description: The Clinical COPD Questionnaire (CCQ) is a 10-item scale where the participant chooses responses on a 0-6 scale for each item. 0 represents no symptoms and 6 represents severe symptoms. The mean of the responses is the final score and ranges from 0-6, 0 is least severe symptoms and 6 is most severe symptoms.
[2]
Measure Analysis Population Description: 40 patients in the sample were diagnosed with interstitial lung disease (ILD)
1.Primary Outcome
Title Change in FEV1 From Baseline Among COPD Patients
Hide Description Change from baseline (among COPD patients only) as measured by pulmonary function testing/spirometry at baseline and again at 6 months post- treatment
Time Frame Measurements pre-treatment and then at 6 months post- treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
All patients with COPD were asked to provide a 6 month post-treatment pulmonary function test. 5 participants completed and returned the test.
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description:

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Overall Number of Participants Analyzed 5
Mean (Full Range)
Unit of Measure: percentage of change in FEV1
12.8
(0 to 100)
2.Primary Outcome
Title Number of Participants Reporting an Improvement in 3 Month-Post Treatment QOL Scores, All Diagnoses
Hide Description Number of patients in the total sample who reported an improved perceived quality of life 3 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame Measurements pre-treatment and then at 3 months post-treatment for all diagnoses
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
148 of 207 enrolled participants completed the 3 month post-treatment QOL survey
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description:

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Overall Number of Participants Analyzed 148
Measure Type: Count of Participants
Unit of Measure: Participants
121
  81.8%
3.Primary Outcome
Title Number of Participants Reporting an Improvement in 6 Month-Post Treatment QOL Scores, All Diagnoses
Hide Description Number of patients in the total sample who reported an improved perceived quality of life 6 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame Measurements pre-treatment and then at 6 months post-treatment among all diagnoses
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
124 patients completed a 6 month post-treatment QOL survey
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description:

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Overall Number of Participants Analyzed 124
Measure Type: Count of Participants
Unit of Measure: Participants
90
  72.6%
4.Other Pre-specified Outcome
Title Number of Participants With COPD Reporting an Improvement in 3 Month-Post Treatment QOL Scores
Hide Description Number of patients with a diagnosis of COPD who reported an improved perceived quality of life 3 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame Measurements for COPD pre-treatment and then at 3 months post-treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
120 patients with COPD completed a 3 month post-treatment QOL survey
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description:

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Overall Number of Participants Analyzed 120
Measure Type: Count of Participants
Unit of Measure: Participants
102
  85.0%
5.Other Pre-specified Outcome
Title Number of Participants With COPD Reporting an Improvement in 6 Month-Post Treatment QOL Scores
Hide Description Number of patients with a diagnosis of COPD who reported an improved perceived quality of life 6 months post-treatment as measured by the Clinical COPD Questionnaire (CCQ).
Time Frame Measurements for COPD pre-treatment and then at 6 months post-treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
101 patients with COPD completed a 6 month post-treatment QOL survey
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description:

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Overall Number of Participants Analyzed 101
Measure Type: Count of Participants
Unit of Measure: Participants
79
  78.2%
6.Other Pre-specified Outcome
Title Number of Participants With Interstitial Lung Disease Reporting an Improvement in 3 Month-Post Treatment QOL Scores
Time Frame Measurements for ILD pre-treatment and then at 3 months post-treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
28 patients with ILD completed a 3 month post-treatment QOL survey
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description:

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Overall Number of Participants Analyzed 28
Measure Type: Count of Participants
Unit of Measure: Participants
20
  71.4%
7.Other Pre-specified Outcome
Title Number of Participants With Interstitial Lung Disease Reporting an Improvement in 6 Month-Post Treatment QOL Scores
Time Frame Measurements for ILD pre-treatment and then at 6 months post-treatment
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
23 patients with ILD completed a 6 month post-treatment QOL survey
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description:

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

Overall Number of Participants Analyzed 23
Measure Type: Count of Participants
Unit of Measure: Participants
13
  56.5%
Time Frame Adverse events monitoring and reporting was done over the entire length of the study, approximately 11 months from March 2016 through December 2016.
Adverse Event Reporting Description Any and all adverse events were reported to the Principal Investigator and reported to MaGil IRB and the supporting organization's quality and compliance monitor within 48 hours of the event.
 
Arm/Group Title Cell Therapy Treated
Hide Arm/Group Description

All patients/participants enrolled will undergo cell therapy

Cell therapy: Each participant will receive cell therapy with stem cells harvested either from peripheral blood or from bone marrow plus peripheral blood; after harvest, cells are processed and returned to the patient via peripheral circulation on the same day.

All-Cause Mortality
Cell Therapy Treated
Affected / at Risk (%)
Total   4/207 (1.93%)    
Show Serious Adverse Events Hide Serious Adverse Events
Cell Therapy Treated
Affected / at Risk (%) # Events
Total   0/207 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0.2%
Cell Therapy Treated
Affected / at Risk (%) # Events
Total   10/207 (4.83%)    
Cardiac disorders   
Bradycardia, not procedure related  1 [1]  1/207 (0.48%)  1
Hypotension  2 [2]  2/207 (0.97%)  2
Surgical and medical procedures   
Dizziness  [3]  7/207 (3.38%)  7
1
Term from vocabulary, Bradycardia
2
Term from vocabulary, Hypotension
Indicates events were collected by systematic assessment
[1]
Participant presented with pre-treatment significant bradycardia and was transported to the emergency department for evaluation. Treatment protocol stopped pending evaluation.
[2]
Participants experienced low blood pressure following bone marrow harvesting of stem cells. Blood pressure was immediately corrected by the participant laying down and receiving intravenous fluids.
[3]
Participant reported dizziness following collection of blood sample for venous cell therapy.
This study is limited by a small sample size for post-treatment pulmonary function test returns and the self-reporting of quality of life scores. The CCQ measure quantifiable and can show trends over time, but it remains a subjective measure.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Melissa Rubio, PhD, APRN
Organization: Lung Institute
Phone: 214-504-2117
Responsible Party: Melissa Rubio, PhD, APRN, Lung Institute
ClinicalTrials.gov Identifier: NCT03044431     History of Changes
Other Study ID Numbers: LI001
First Submitted: February 3, 2017
First Posted: February 7, 2017
Results First Submitted: June 27, 2017
Results First Posted: October 25, 2017
Last Update Posted: October 15, 2018